Hematopoietic stem: Cell transplantation for severe comibined immunodeficiency disease

Immunology and Allergy Clinics of North America

Volumn 20, Issue 1, 2000, Pages 207-220

  Small, Trudy ^b  

^a MEMORIAL SLOAN KETTERING CANCER CENTER   (United States)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

CD4 ANTIGEN; CYCLOSPORIN A; METHOTREXATE; METHYLPREDNISOLONE;

BONE MARROW TRANSPLANTATION; COMBINED IMMUNODEFICIENCY; GENE THERAPY; GRAFT FAILURE; GRAFT VERSUS HOST REACTION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HLA MATCHING; HUMAN; PRIORITY JOURNAL; REVIEW; UMBILICAL CORD BLOOD;

EID: 0034001148     PISSN: 08898561     EISSN: None     Source Type: Journal    
DOI: 10.1016/S0889-8561(05)70142-9     Document Type: Article

References (88)

1. F.S. Rosen M.D. Cooper R.J.P. Wedgewood The primary immunodeficiencies N Engl J Med 333 1995 431 440
2. F.S. Rosen F. Aiuti W. Hitzig Meeting report: Primary immunodeficiency disease Clin Immunol Immunopathol 28 1983 450 475
3. E.R. Giblett J.E. Anderson F. Cohen Adenosine deaminase deficiency in two patients with severely impaired cellular immunity Lancet 2 1972 1067 1069
4. M.S. Hershfield Adenosine deaminase deficiency: Clinical expression, molecular basis, and therapy Semin Hematol 35 1998 291 293
5. G. Omenn Familial reticuloendotheliosis N Engl J Med 273 1965 427 432
6. 6+ Langerhans-like cells Virchow's Archive 407 1985 69 82
7. O.M. DeVaal V. Seynhaeve Reticular dysgenesis Lancet 2 1959 1123 1125
8. T.N. Small D.A. Wall J. Kurtzberg Association of reticular dysgenesis (thymic alymphoplasia and congenital aleukocytosis) with bilateral sensorineural deafness J Pediatr 135 1999 387 389
9. R.A. Gatti N. Platt H.H. Pomerance Hereditary lymphopenic agammaglobulinemia associated with a distinctive form of short-limbed dwarfism and ectodermal dysplasia J Pediatr 75 1969 675 684
10. E.W. Gelfand J.M. Oliver R.K. Schuurman Abnormal lymphocyte capping in a patient with severe combined immunodeficiency disease N Engl J Med 301 1979 1245 1249
11. L.K.L. Jung S.M. Fu T. Hara Defective expression of T-cell associated glycoprotein in severe combined immunodeficiency J Clin Invest 77 1986 940 946
12. B. Alarcon J.R. Regueiro A. Arnaiz-Villena Familial defect in the surface expression of the T-cell receptor-CD3 complex N Engl J Med 319 1988 1203 1208
13. C.M. Roifman D. Hummel H. Martinez-Valdez Depletion of CD8+ cells in human thymic medulla results in selective immune deficiency J Exp Med 170 1989 2177 2182
14. W.J. Monafo S.H. Polmar S. Neudorf A hereditary immunodeficiency characterized by CD8+ T lymphocyte deficiency and impaired lymphocyte activation Clin Exp Immunol 90 1992 390 393
15. A. Fischer P. Landais W. Friedrich European experience of bone-marrow transplantation for severe combined immunodeficiency Lancet 336 1990 850 854
16. R.H. Buckley R.I. Schiff S.E. Schiff Human severe combined immunodeficiency: Genetic, phenotypic, and functional diversity in 108 infants J Pediatr 130 1997 378 87
17. Y. Dror R.M. Gallagher D.W. Wara Immune reconstitution in severe combined immunodeficiency disease after lectin-treated, TCD haploidentical bone marrow transplantation Blood 1993 2021 2030
18. R.J. O'Reilly W. Friedrich T.N. Small Hematopoietic cell transplantation for immunodeficiency disorders Thomas E.D. Blume K.G. Forman S.J. Hematopoietic Cell Transplantation ed 2 1999 Blackwell Science Malden, MA 1154 1172
19. R. Parkman E.W. Gelfand F.S. Rosen Severe combined immunodeficiency and adenosine deaminase deficiency N Engl J Med 14 1975 714 719
20. D.R. Ownby S. Pizzo L. Blackmon Severe combined immunodeficiency with leukopenia (reticular dysgenesis) in siblings: Immunologic and histopathologic findings J Pediatr 89 1976 382 387
21. T. Dooley L.D. Fairbanks H.A. Simmonds First trimester diagnosis of adenosine deaminase deficiency Prenat Diagn 7 1987 561 565
22. A. Durandy Y. Dumez D. Guy-Grand Prenatal diagnosis of severe combined immunodeficiency disease J Pediatr 101 1982 995 997
23. M. Noguchi H. Yi H.M. Rosenblatt Interleukin-2 receptor γ chain mutation results in X-linked severe combined immunodeficiency in humans Cell 73 1993 147 57
24. J.M. Puck S.M. Deschenes J.C. Porter The interleukin-2 receptor γ chain maps to Xq13.1 and is mutated in X-linked severe combined immunodeficiency, SCIDX1 Hum Mol Genet 2 1993 1099 104
25. J.M. Puck A.E. Pepper P.S. Henthorn Mutation analysis of IL2RG in human X-linked severe combined immunodeficiency Blood 89 1997 1968 77
26. S.M. Russell N. Tayebi H. Nakajima Mutation of Jak3 in a patient with SCID: Essential role of Jak3 in lymphoid development Science 270 1995 797 800
27. P. Macchi A. Villa S. Gillani Mutations of Jak-3 gene in patients with autosomal severe combined immunodeficiency disease (SCID) Nature 377 1995 65 68
28. K. Schwarz G.H. Gauss L. Ludwig RAG mutations in human B cell–negative SCID Science 274 1996 97 99
29. P. Mombaerts J. Iacomini R.S. Johnson RAG-1–deficient mice have no mature B and T lymphocytes Cell 68 1992 869 877
30. A. Villa S. Santagata F. Bozzi Partial V(D)J recombination activity leads to Omenn syndrome Cell 93 1998 885 896
31. A. Villa S. Santagata F. Bozzi Omenn syndrome: A disorder of Rag1 and Rag2 genes J Clin Immunol 19 1999 87 97
32. E. Arpaia M. Shahar H. Dadi Defective T-cell–receptor signaling and CD8+ thymic selection in humans lacking Zap-70 kinase Cell 76 1994 947 958
33. M.E. Elder D. Lin J. Clever Human severe combined immunodeficiency due to a defect in ZAP-70, a T-cell tyrosine kinase Science 264 1994 1596 1599
34. A.C. Chan T.A. Kadlecek M.E. Elder ZAP-70 deficiency in an autosomal recessive form of severe combined immunodeficiency Science 264 1994 1599 1601
35. J.M. Puck L. Middelton A.E. Pepper Carrier and prenatal diagnosis of X-linked severe combined immunodeficiency: Mutation detection methods and utilization Hum Genet 1997 628 633
36. R.F. Schumacher P. Mella F. Lalatta Prenatal diagnosis of JAK3 deficient SCID Prenat Diagn 19 1999 653 656
37. R.A. Gatti H.J. Meeuwissen H.D. Allen Immunological reconstitution of sex-linked lymphopenic immunological deficiency Lancet 2 1968 1366 1369
38. R.H. Buckley Bone marrow reconstitution in primary immunodeficiency Clinical Immunology: Principles and Practice 2 1995 1813 1830
39. A.H. Filipovich Stem cell transplantation from unrelated donors for correction of primary immunodeficiencies Organ and bone marrow transplantation 16 1996 393 377
40. P. Rubinstein C. Carrier A. Scaradavou Outcomes among 562 recipients of placental-blood transplants from unrelated donors N Engl J Med 339 1998 1565 1577
41. A.W. Flake E.D. Zanjani In utero hematopoietic stem cell transplantation: Ontogenic opportunities and biologic barriers Blood 94 1999 2179 2191
42. R.H. Buckley S.E. Schiff R.I. Schiff Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency N Engl J Med 340 1999 508 516
43. J.L. Stephan V. Vlekova Le Deist F. Severe combined immunodeficiency: A retrospective single-center study of clinical presentation and outcome in 117 patients J Pediatr 123 1993 564 572
44. L.J. Sindel R.H. Buckley S.E. Schiff Severe combined immunodeficiency with natural killer cell predominance: Abrogation of graft-versus-host disease and immunologic reconstitution with HLA-identical bone marrow cells J Allergy Clin Immunol 73 1984 829 836
45. A.B. Kenny W.H. Hitzig Bone marrow transplantation for severe combined immunodeficiency Eur J Pediatr 131 1979 155 177
46. M. Boeckh R.A. Bowden T. Gooley Successful modification of a pp65 antigenemia-based early treatment strategy for prevention of cytomegalovirus disease in allogeneic marrow transplant recipients Blood 93 1999 1781 1782
47. van der Meer J.T. W.L. Drew R.A. Bowden Summary of the International Consensus Symposium on Advances in the Diagnosis, Treatment and Prophylaxis and Cytomegalovirus Infection Antiviral Res 32 1996 119 140
48. J.A. Englund E. Whimbey R.L. Atmar Diagnosis of respiratory viruses in cancer and transplant patients Curr Clin Top Infect Dis 19 1999 30 59
49. R.J. Leggiardo J.A. Winkelstein W.T. Hughes Prevalence of Pneumocystis carinii pneumonitis in severe combined immunodeficiency J Pediatr 99 1981 96 98
50. C.H. Wendt M.I. Hertz Respiratory syncytial virus and parainfluenza virus infections in the immunocompromised host Semin Respir Infect 10 1995 224 231
51. W.T. Hughes Current issues in the epidemiology, transmission, and reactivation of Pneumocystis carinii Semin Respir Infect 13 1998 283 288
52. van Burik J.H. W. Leisenring D. Myerson The effect of prophylactic fluconazole on the clinical spectrum of fungal diseases in bone marrow transplant recipients with special attention to hepatic candidiasis: An autopsy study of 355 patients Medicine 77 1998 246 254
53. H. Henry Balfour: Drug Therapy: Antiviral drugs N Engl J Med 1999 340
54. R.J. O'Reilly N. Kapoor D. Kirkpatrick Transplantation of hematopoetic cells for lethal congenital immunodeficiencies Birth Defects 19 1983 129 137
55. von Boehmer H. J. Sprent M. Nabholz Tolerance to histocompatibility determinants in tetraparental bone marrow chimeras J Exp Med 141 1975 322 334
56. N.A. Kernan T-cell depletion for the prevention of graft-versus-host disease Thomas E.D. Blume K.G. Forman S.J. Hematopoietic Cell Transplantation ed 2 1999 Blackwell Science Malden, MA 186 196
57. W. Friedrich S.F. Goldmann W. Ebell Severe combined immunodeficiency: Treatment by bone marrow transplantation in 15 infants using HLA-haploidentical donors Eur J Pediatr 144 1985 125 130
58. M.J. Cowan D.W. Wara P.S. Weintrub Haploidentical bone marrow transplantation for severe combined immunodeficiency disease using soybean agglutinin-negative, T-depleted marrow grafts J Clin Immunol 5 1985 370 376
59. R. Blütters-Sawatzki W. Friedrich W. Ebell HLA-haploidentical bone marrow transplantation in three infants with adenosine deaminase deficiency: Stable immunological reconstitution and reversal of skeletal abnormalities Eur J Pediatr 149 1989 104 109
60. A. Fischer A. Durandy De Villartay J.P. HLA-haploidentical bone marrow transplantation for severe combined immunodeficiency using E-rosette fractionation and cyclosporine Blood 67 1986 444 449
61. E. Reinherz R. Geha J.M. Rappeport Reconstitution after transplantation with T-lymphocyte–depleted HLA haplotype-mismatched bone marrow for severe combined immunodeficiency Proc Natl Acad Sci USA 79 1982 6047 6051
62. J. Ritz Use of monoclonal antibodies in autologous and allogeneic bone marrow transplantation Clin Hematol 12 1983 813 832
63. A.R. Hayward S. Murphy J. Githens Failure of a panreactive anti–T-cell antibody, OKT3, to prevent graft versus host disease in severe combined immunodeficiency J Pediatr 100 1982 665 668
64. M.E. Trigg R. Billing P.M. Sondel Clinical trial depleting T lymphocytes from donor marrow for matched and mismatched allogeneic bone marrow transplants Cancer Treat Rep 69 1985 377 386
65. R.C. Moen S.D. Horowitz P.M. Sondel Immunologic reconstitution after haploidentical bone marrow transplantation for immune deficiency disorders: Treatment of bone marrow cells with monoclonal antibody CT-2 and complement Blood 70 1987 664 669
66. G. Morgan D.C. Linen L.T. Knott Successful haploidentical mismatched bone marrow transplantation in severe combined immunodeficiency: T-cell removal using CAMPATH-1 monoclonal antibody and E-rosetting Br J Haematol 62 1986 421 430
67. A.M. Dickinson M.M. Reid M. Abunum In vitro T-cell depletion using Campath 1M for mismatched BMT for severe combined immunodeficiency (SCID) Bone Marrow Transplantation 19 1997 323 329
68. M.P. Martin-Hernandez R. Arrieta A. Martinez Haploidentical peripheral blood stem cell transplantation with a combination of CD34 selection and T-cell depletion as graft-versus-host disease prophylaxis in a patient with severe combined immunodeficiency Bone Marrow Transplant 20 1997 797 799
69. Y. Reisner N. Kapoor D. Kirkpatrick Transplantation for severe combined immunodeficiency HLA-A, B, D, Dr incompatible parental marrow fractionated by soybean agglutinin and sheep red blood cells Blood 61 1983 341 348
70. R.J. O'Reilly N. Kapoor D. Kirkpatrick Transplantation for severe combined immunodeficiency using histoincompatible parental marrow fractionated by soybean agglutinin and sheep red blood cells: Experience in six consecutive cases Transplant Proc 17 1983 455 459
71. E. Haddad P. Landais W. Friedrich Long-term immune reconstitution and outcome after HLA-nonidentical T-cell–depleted bone marrow transplantation for severe combined immunodeficiency: A European retrospective study of 116 patients Blood 91 1998 3646 3653
72. Y. Bertrand P. Landais W. Friedrich Influence of severe combined immunodeficiency phenotype on the outcome of HLA nonidentical T-cell–depleted bone marrow transplantation J Pediatr 134 1999 740 748
73. R.J. O'Reilly B. Dupont S. Pahwa Reconstitution in severe combined immunodeficiency by transplantation of marrow from an unrelated donor N Engl J Med 297 1977 1311 1318
74. A.H. Filipovich R.S. Shapiro N.K.C. Ramsay Unrelated donor bone marrow transplantation for correction of lethal congenital immunodeficiencies Blood 80 1992 270 276
75. A.H. Filipovich Bone marrow transplantation from unrelated donors for congenital immunodeficiencies Bone Marrow Transplant 11 suppl 1 1993 78 80
76. A.K. Junker K.J. Chan B.G. Massing Clinical and immune recovery from Omenn syndrome after bone marrow transplantation J Pediatr 114 1989 596 600
77. Dalal I, Reid B, Doyle J, et al: Matched unrelated bone marrow transplantation for combined immunodeficiency. Bone Marrow Transplant, in press
78. E. Gluckman V. Rocha A. Boyer-Chammard Outcome of cord-blood transplantation from related and unrelated donors N Engl J Med 337 1997 373 382
79. J. Kurtzberg M. Laughlin M. Graham Placental blood as a source of hematopoietic stem cells for transplantation into unrelated recipients N Engl J Med 335 1996 157 166
80. J. Wagner J. Rosenthal R. Sweetman Successful transplantation of HLA-matched and HLA-mismatched umbilical cord blood from unrelated donors: Analysis of engraftment and acute graft-versus-host disease Blood 88 1996 795 802
81. J.E. Wagner N.A. Kernan M. Steinbuch Allogeneic sibling umbilical-cord-blood transplantation in children with malignant and nonmalignant disease Lancet 1995 214 219
82. A.P. Knutsen D.A. Wall Kinetics of T-cell development of umbilical cord blood transplantation in severe T-cell immunodeficiency disorders J Allergy Clin Immunol 103 part 1 1999 823 832
83. A. Benito M.A. Diaz F. Alonso Successful unrelated umbilical cord blood transplantation in a child with Omenn's syndrome Pediatr Hematol Oncol 16 1999 361 366
84. N. Kapoor L.K.L. Jung D. Engelhard Lymphoma in a patient with severe combined immunodeficiency with adenosine deaminase deficiency, following unsustained engraftment of histoincompatible TCD bone marrow J Pediatr 100 1986 435 438
85. W.T. Shearer J. Ritz M.J. Finegold Epstein-Barr virus–associated B-cell proliferations of diverse clonal origins after bone marrow transplantation in a 12-year-old patient with severe combined immunodeficiency N Engl J Med 312 1985 1151 1159
86. B.D. Spector G.S. Perry J.H. Kersey Genetically determined immunodeficiency diseases (SCID) and malignancy: Report from the immunodeficiency cancer registry Clin Immunol Immunopathol 11 1978 12 29
87. A.W. Flake E.D. Zanjani In utero hematopoietic stem cell transplantation: Ontogenic opportunities and biologic barriers Blood 94 1999 2179 2191
88. K.I. Weinberg D.B. Kohn Gene therapy for congenital lymphoid immunodeficiency diseases Semin Hematol 35 1998 354 366